Table 2.
Overview of Included Trials and Abstracts
| 1st Author | Country | Year | Journal | Trial name | Trial phase | Tumor type | |
|---|---|---|---|---|---|---|---|
| 1 | Sridharan Gururangan | USA | 2002 | Journal of Clinical Oncology | Carboplatin in children with progressive low-grade gliomas | II | LGG |
| 2 | Mark T Jennings | USA | 2002 | Journal of Clinical Oncology | Preradiation chemotherapy in primary high-risk brainstem tumors | II | Brainstem gliomas |
| 3 | Stewart J Kellie | Australia | 2002 | Medical and Pediatric Oncology | Activity of postoperative carboplatin, etoposide, and high-dose methotrexate in pediatric CNS embryonal tumors | II | CNS embyronal tumors |
| 4 | L S Lashford | UK | 2002 | Journal of Clinical Oncology | Temozolomide in malignant gliomas of childhood | II | Malignant glioma |
| 5 | Thomas E Merchant | USA | 2002 | International Journal of Radiation Oncology Biology Physics | Conformal radiation therapy for pediatric patients with localized low-grade astrocytoma and ependymoma | II | Low-grade astrocytoma and ependymoma |
| 6 | Thomas E Merchant | USA | 2004 | Journal of Clinical Oncology | Conformal radiation therapy and evaluation of radiation-related CNS effects for pediatric patients with localized ependymoma | II | Localized ependymoma |
| 7 | Stanislaw R Burzynski | USA | 2005 | Integrative Cancer Therapies | Long-term survival of high-risk pediatric patients with primitive neuroectodermal tumors treated with antineoplastons A10 and AS2-1 | II | Primitive neuroectodermal tumors |
| 8 | Tobey J MacDonald | USA | 2005 | Cancer | High-dose chemotherapy before radiation in children with newly diagnosed high-grade astrocytoma | II | High-grade astrocytoma |
| 9 | Murali M Chintagumpala | USA | 2006 | Journal of Neuro-Oncology | Procarbazine and topotecan in children with high-grade glioma | II | HGG |
| 10 | Maryam Fouladi | USA | 2006 | Cancer | Oxaliplatin in children with recurrent or refractory medulloblastoma, supratentorial primitive neuroectodermal tumors, and atypical teratoid rhabdoid tumors | II | Recurrent or refractory medulloblastoma, supratentorial primitive Neuroectodermal tumors, and atypical teratoid rhabdoid tumors |
| 11 | Katherine Warren | USA | 2006 | Cancer Chemotherapy and Pharmacology | Intravenous lobradimil and carboplatin in childhood brain tumors | II | Brainstem glioma, high-grade glioma, low-grade glioma, 1medullobastoma/primitive neuroectodermal tumor (PNET), and ependymoma |
| 12 | Maryam Fouladi | USA | 2007 | Cancer | Farnesyl transferase inhibitor, tipifarnib, in children with recurrent or progressive high-grade glioma, medulloblastoma/primitive neuroectodermal tumor, or brainstem glioma | II | recurrent or progressive high-grade glioma, medulloblastoma/primitive neuroectodermal tumor, or brainstem glioma |
| 13 | H Stacy Nicholson | USA | 2007 | Cancer | Temozolomide in children and adolescents with recurrent central nervous system tumors | II | CNS tumors—mainly high/low grade astrocytoma, medulloblastoma, and PNET |
| 14 | David N Korones | USA | 2008 | Pediatric Blood & Cancer | Treatment of children with diffuse intrinsic brain stem glioma with radiotherapy, vincristine, and oral VP-16 | II | DIPG |
| 15 | Jeffrey Allen | USA | 2009 | International Journal of Radiation Oncology Biology Physics | Preradiotherapy chemotherapy followed by hyperfractionated radiotherapy for newly diagnosed high-risk medulloblastoma/primitive neuroectodermal tumor | II | Medulloblastoma/primitive neuroectodermal tumor |
| 16 | Sylvain Baruchel | Canada | 2009 | European Journal of Cancer | Imatinib in recurrent and refractory pediatric central nervous system tumors | II | CNS tumors—mainly astrocytoma, ependymoma, GBM, medulloblastoma |
| 17 | Sridharan Gururangan | USA | 2010 | Journal of Clinical Oncology | Lack of efficacy of bevacizumab plus irinotecan in children with recurrent malignant glioma and diffuse brainstem glioma | II | Recurrent malignant glioma and diffuse brainstem glioma |
| 18 | Daphne A. Haas-Kogan | USA | 2011 | Neuro-oncology | Tipifarnib and radiation in children with newly diagnosed diffuse intrinsic pontine gliomas | II | DIPG |
| 19 | Jane E Minturn | USA | 2011 | Pediatric Blood & Cancer | Metronomic oral topotecan for recurrent childhood brain tumors | II | CNS tumors—mainly ependymoma, high-grade glioma (HGG), brainstem glioma, and primitive neuroectodermal tumor |
| 20 | Ian F Pollack | USA | 2011 | Neuro-oncology | Gefitinib and irradiation in children with newly diagnosed brainstem gliomas | II | Diffuse intrinsic brainstem glioma. |
| 21 | James H Garvin Jr | USA | 2012 | Pediatric Blood & Cancer | Pre-irradiation chemotherapy for childhood intracranial ependymoma | II | Intracranial ependymoma |
| 22 | Birgit Geoerger | France | 2012 | European Journal of Cancer | Temsirolimus in children with high-grade glioma, neuroblastoma and rhabdomyosarcoma | II | High-grade glioma, neuroblastoma and rhabdomyosarcoma |
| 23 | Katherine Warren | USA | 2012 | Cancer | Study of pegylated interferon α-2b (PEG-Intron(®)) in children with diffuse intrinsic pontine glioma | II | Diffuse intrinsic pontine glioma |
| 24 | Katherine Warren | USA | 2012 | Journal of Neuro-Oncology | Study of O6-benzylguanine and temozolomide in pediatric patients with recurrent or progressive high-grade gliomas and brainstem gliomas | II | High-grade gliomas and brainstem gliomas |
| 25 | Kristin A Bradley | USA | 2013 | International Journal of Radiation Oncology Biology Physics | Motexafin-gadolinium and involved field radiation therapy for intrinsic pontine glioma of childhood | II | Intrinsic pontine glioma |
| 26 | Jacques Grill | France | 2013 | Neuro-oncology | Irinotecan in combination with temozolomide (TEMIRI) in children with recurrent or refractory medulloblastoma | II | Medulloblastoma |
| 27 | Darren Hargrave | UK | 2013 | Journal of Neuro-Oncology | Irinotecan in combination with temozolomide (TEMIRI) in children with newly diagnosed high-grade glioma | II | HGG |
| 28 | Tobey J MacDonald | USA | 2013 | Neuro-oncology | Cilengitide in the treatment of refractory or relapsed high-grade gliomas in children | II | HGG |
| 29 | Anne B Warwick | USA | 2013 | Pediatric Blood & Cancer | Pemetrexed in children and adolescents with refractory solid tumors | II | Refractory solid tumors including ependymoma, Ewing sarcoma, medulloblastoma, neuroblastoma, osteosarcoma |
| 30 | Angela Di Giannatale | France | 2014 | European Journal of Cancer | Temozolomide in combination with topotecan (TOTEM) in relapsed or refractory neuroblastoma | II | Relapsed or refractory neuroblastoma |
| 31 | Matthias A Karajannis | USA | 2014 | Neuro-oncology | Sorafenib in children with recurrent or progressive low-grade astrocytomas | II | Recurrent or progressive low-grade astrocytomas |
| 32 | C. Kretschmar | USA | 2014 | Pediatric Blood & Cancer | Pre-radiation chemotherapy with response-based Radiation therapy in children with central nervous system germ cell tumors | II | Germ cell tumors—mainly germinoma |
| 33 | Maryam Fouladi | USA | 2014 | Journal of Neuro-Oncology | A molecular biology and phase II trial of lapatinib in children with refractory CNS malignancies: a pediatric brain tumor consortium study | II | CNS tumors—medulloblastoma, HGG, ependymoma |
| 34 | Ute Bartels | Canada | 2014 | Neuro-oncology | Phase 2 study of safety and efficacy of nimotuzumab in pediatric patients with progressive diffuse intrinsic pontine glioma | II | DIPG |
| 35 | Stanislaw R Burzynski | USA | 2014 | Child’s Nervous System | The response and survival of children with recurrent diffuse intrinsic pontine glioma based on phase II study of antineoplastons A10 and AS2-1 in patients with brainstem glioma | II | Recurrent pediatric diffuse intrinsic pontine gliomas (RPDIPG) |
| 36 | Graziella Cefalo | Italy | 2014 | Neuro-oncology | Temozolomide is an active agent in children with recurrent medulloblastoma/primitive neuroectodermal tumor: an Italian multi-institutional phase II trial | II | Recurrent medulloblastoma/primitive neuroectodermal tumor |
| 37 | Maura Massimino | Italy | 2014 | Journal of Neuro-Oncology | Results of nimotuzumab and vinorelbine, radiation and re-irradiation for diffuse pontine glioma in childhood | II | DIPG |
| 38 | Tamara Vern-Gross | USA | 2014 | Neuro-Oncology | Prospective evaluation of local control and late effects of conformal radiation therapy in children, adolescents, and young adults with high-grade glioma | II | HGG |
| 39 | Nathan J Robison | USA | 2014 | Pediatric Blood & Cancer | A phase II trial of a multi-agent oral antiangiogenic (metronomic) regimen in children with recurrent or progressive cancer | II | HGG, ependymoma, LGG, medulloblastoma/PNET, neuroblastoma |
| 40 | Patricia Robinson | USA | 2014 | Journal of Neuro-Oncology | Multimodality therapy for CNS mixed malignant germ cell tumors (MMGCT): Results of a phase II multi-institutional study | II | CNS mixed malignant germ cell tumors (MMGCT) |
| 41 | Ibrahim Qaddoumi | USA | 2014 | Frontiers in Oncology | Phase II trial of erlotinib during and after radiotherapy in children with newly diagnosed high-grade gliomas | II | HGG (AA and GBM) |
| 42 | Diana Osorio | USA | 2014 | Journal of Neuro-Oncology | Pre-irradiation intensive induction and marrow-ablative consolidation chemotherapy in young children with newly diagnosed high-grade brainstem gliomas: report of the “head-start” I and II clinical trials | II | High-grade brainstem glioma |
| 43 | Orren Beaty | USA | 2015 | Pediatric Blood & Cancer | A Phase II trial and pharmacokinetic study of oxaliplatin in children with refractory solid tumors: a children’s oncology group study | II | Ewing sarcoma/peripheral PNET, osteosarcoma, rhabdomyosarcoma, neuroblastoma, high- and low-grade astrocytoma, brain stem glioma, ependymoma, hepatoblastoma and selected rare tumors |
| 44 | Bruce Cohen | USA | 2015 | Pediatric Neurology | Pilot study of intensive chemotherapy with peripheral hematopoietic cell support for children <3 years of age with malignant brain tumors, the ccg-99703 phase I/II study. a report from the children’s oncology group | II | CNS tumors—medulloblastoma, supratentorial PNET, ependymoma |
| 45 | Mariko DeWire | USA | 2015 | Journal of Neuro-Oncology | An open-label, 2-stage, phase II study of bevacizumab and lapatinib in children with recurrent or refractory ependymoma: a collaborative ependymoma research network study (CERN) | II | Recurrent or refractory ependymoma |
| 46 | Stewart Goldman | USA | 2015 | Journal of Clinical Oncology | Phase II trial assessing the ability of neoadjuvant chemotherapy with or without second-look surgery to eliminate measurable disease for nongerminomatous germ cell tumors: a children’s oncology group study | II | Nongerminomatous GCTs |
| 47 | Lisa L R Hartman | USA | 2015 | Journal of Pediatric Hematology/Oncology | Pediatric phase II trials of poly-ICLC in the management of newly diagnosed and recurrent brain tumors | II | LGG |
| 48 | Regina Jakacki | USA | 2016 | Neuro-oncology | Phase 2 study of concurrent radiotherapy and temozolomide followed by temozolomide and lomustine in the treatment of children with high-grade glioma: a report of the Children’s Oncology Group ACNS0423 study | II | HGG |
| 49 | Regina Jakacki | USA | 2016 | Journal of Neuro-Oncology | Single-agent erlotinib versus oral etoposide in patients with recurrent or refractory pediatric ependymoma: a randomized open-label study | II | Ependymoma |
| 50 | Alvaro Lassaletta | Canada | 2016 | Journal of Clinical Oncology | Phase II weekly vinblastine for chemotherapy-naïve children with progressive low-grade glioma: A Canadian pediatric brain tumor consortium study | II | LGG |
| 51 | Torunn Yock | USA | 2016 | The Lancet Oncology | Long-term toxic effects of proton radiotherapy for pediatric medulloblastoma: A phase 2 single-arm study | II | Medulloblastoma |
| 52 | Cynthia Wetmore | USA | 2016 | Cancer Medicine | Phase II evaluation of sunitinib in the treatment of recurrent or refractory high-grade glioma or ependymoma in children: a children’s Oncology Group Study ACNS1021 | II | HGG and ependymoma |
| 53 | Ralph Salloum | USA | 2016 | Journal of Neuro-Oncology | A molecular biology and phase II study of imetelstat (GRN163L) in children with recurrent or refractory central nervous system malignancies: a pediatric brain tumor consortium study | II | Medulloblastoma, high-grade glioma (HGG) and ependymoma |
| 54 | John Lucas Jr | USA | 2017 | International Journal of Radiation Oncology Biology Physics | Prognostic relevance of treatment failure patterns in pediatric high-grade glioma: Is there a role for a revised failure classification system? | II | HGG |
| 55 | Margaret Macy | USA | 2017 | Pediatric Blood & Cancer | A pediatric trial of radiation/cetuximab followed by irinotecan/cetuximab in newly diagnosed diffuse pontine gliomas and high-grade astrocytomas: A Pediatric Oncology Experimental Therapeutics Investigators’ Consortium study | II | DIPG and high-grade astrocytoma |
| 56 | Jacques Grill | France | 2018 | Journal of Clinical Oncology | Phase II, open-label, randomized, multicenter trial (HERBY) of bevacizumab in pediatric patients with newly diagnosed high-grade glioma | II | HGG |
| 57 | Peter Manley | USA | 2018 | Pediatric Blood & Cancer | A phase 1/2 dose-finding, safety, and activity study of cabazitaxel in pediatric patients with refractory solid tumors including tumors of the central nervous system | II | Recurrent HGG and DIPG |
| 58 | Yousra Izzuddeen | India | 2019 | Journal of Neuro-Oncology | Hypofractionated radiotherapy with temozolomide in diffuse intrinsic pontine gliomas: a randomized controlled trial | II | DIPG |
| 59 | Joel Cherlow | USA | 2019 | International Journal of Radiation Oncology Biology Physics | Conformal radiation therapy for pediatric patients with low-grade glioma: results from the children’s oncology group phase 2 study ACNS0221 | II | LGG |
| 60 | Jason Fangusaro | USA | 2019 | The Lancet Oncology | Selumetinib in pediatric patients with BRAF-aberrant or neurofibromatosis type 1-associated recurrent, refractory, or progressive low-grade glioma: a multicentre, phase 2 trial | II | BRAF-aberrant or neurofibromatosis type 1-associated recurrent, refractory, or progressive LGG |
| 61 | Jason Fangusaro | USA | 2019 | Journal of Clinical Oncology | Phase II trial of response-based radiation therapy for patients with localized CNS nongerminomatous germ cell tumors: a children’s oncology group study | II | CNS NGGCTs |
| 62 | Darren Hargrave | USA | 2019 | Clinical Cancer Research | Efficacy and safety of dabrafenib in pediatric patients with BRAF V600 mutation–positive relapsed or refractory low-grade glioma: results from a phase I/IIa study | II | LGG |
| 63 | Patricia Baxter | USA | 2020 | Neuro-oncology | A phase I/II study of veliparib (ABT-888) with radiation and temozolomide in newly diagnosed diffuse pontine glioma: A Pediatric Brain Tumor Consortium study | II | DIPG |
| 64 | Stewart Goldman | USA | 2020 | Neuro-oncology | Phase II study of peginterferon alpha-2b for patients with unresectable or recurrent craniopharyngiomas: a Pediatric Brain Tumor Consortium report | II | Craniopharyngioma |
| 65 | Lucie Lafay-Cousin | Canada | 2020 | Journal of Clinical Oncology | Phase II study of nonmetastatic desmoplastic medulloblastoma in children younger than 4 years of age: a Report of the Children’s Oncology Group (ACNS1221) | II | Nodular desmoplastic medulloblastoma (ND) and medulloblastoma with extensive nodularity (MBEN) |
| 66 | Gwénaël Le Teuff | France | 2020 | Pediatric Blood & Cancer | Phase II study of temozolomide and topotecan (TOTEM) in children with relapsed or refractory extracranial and central nervous system tumors including medulloblastoma with post hoc Ba1ian analysis: A European ITCC study | II | CNS tumors—medulloblastoma and PNET expansion |
| 67 | Jack Meng-Fen Su | USA | 2020 | Pediatric Blood & Cancer | A phase 2 study of valproic acid and radiation, followed by maintenance valproic acid and bevacizumab in children with newly diagnosed diffuse intrinsic pontine glioma or high-grade glioma | II | DIPG and HGG |
| 68 | Keiko Okada | Japan | 2020 | Pediatric Blood & Cancer | Phase II study of reduced-dose craniospinal irradiation and combination chemotherapy for children with newly diagnosed medulloblastoma: a report from Japanese Pediatric brain tumor consortium | II | Medulloblastoma |
| 1 | Roger Packer | USA | 2006 | Journal of Clinical Oncology | Phase III study of craniospinal radiation therapy followed by adjuvant chemotherapy for newly diagnosed average-risk medulloblastoma | III | Average-risk medulloblastoma |
| 2 | Birgitta Lannering | Europe | 2012 | Journal of Clinical Oncology | Hyperfractionated vs conventional radiotherapy followed by chemotherapy in standard-risk medulloblastoma: Results from the randomized multicentre HIT-SIOP PNET 4 Trial | III | Medulloblastoma |
| 3 | Nancy Tarbell | USA | 2013 | Journal of Clinical Oncology | High-risk medulloblastoma: a pediatric oncology group randomized trial of chemotherapy before or after radiation therapy (POG 9031) | III | High-risk medulloblastoma |
| 4 | Douglas Strother | USA | 2013 | Neuro-oncology | Benefit from prolonged dose-intensive chemotherapy for infants with malignant brain tumors is restricted to patients with ependymoma: a report of the Pediatric Oncology Group randomized controlled trial 9233/34 | III | Medulloblastoma, ependymoma, supratentorial primitive neuroectodermal tumor (sPNET) and other malignant brain tumors |
| 5 | Mohamed S. Zaghloul | Egypt | 2014 | Radiotherapy & Oncology | Hypofractionated conformal radiotherapy for pediatric diffuse intrinsic pontine glioma (DIPG): A randomized controlled trial | III | DIPG |
| 6 | V Batra | USA | 2015 | Pediatric Blood & Cancer | Long-term survival of children <6 years of age enrolled on the CCG-945 phase III trial for newly-diagnosed high-grade glioma | III | HGG |
| 7 | Rakesh Jalali | India | 2017 | JAMA Oncology | Efficacy of stereotactic conformal radiotherapy vs conventional radiotherapy on benign and low-grade brain tumors: a randomized clinical trial | III | Benign and low-grade tumors |
| 8 | Maria Eveslage | Germany | 2019 | Deutsches Arzteblatt International | The postoperative quality of life in children and adolescents with craniopharyngioma | III | Craniopharyngioma |